Epidiolex Approved for Treatment of Dravet and Lennox-Gastaut Syndromes

Tuesday, June 26, 2018

Yesterday, the Food and Drug Administration (FDA) approved cannabidiol (CBD) oral solution (Epidiolex; GW Research, Carlsbad, CA) for the treatment of seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome (DS), in patients age 2 and up. This is the first FDA-approved drug e derived from marijuana and the first drug approved for the treatment of patients with DS.

CBD is a chemical component of the Cannabis sativa plant, more commonly known as marijuana, that does not cause intoxication or euphoria—the high— that comes from tetrahydrocannabinol (THC). Importantly, CBD is carefully manufactured in a controlled and standardized manner to produce a medicine at a consistent dose for every dose given. It is a pharmaceutical grade formulation that patients can reliably use without concerns of psychoactive effects, addition disorders, or safety concerns associated with uncontrolled products.

In clinical trials, CBD significantly reduced the frequency of drop seizures in patients with LGS by as much as 41.9% compared to a 17.2% drop with placebo and decreased seizure frequency by 39% in patients with DS compared to a 13% decrease with placebo.

As with any FDA-approved drug, CBD must be dispensed information about the drug’s uses and risks. Risks include thoughts about suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression, panic attacks, and elevated liver enzymes. CBD is currently a Schedule I substance because it is a chemical component of the cannabis plant.

FDA commissioner Scott Gottlieb, MD said, “This approval serves as a reminder that advancing sound development programs that properly evaluate active ingredients contained in marijuana can lead to important medical therapies, and the FDA is committed to this kind of careful scientific research and drug development.” Dr. Gottlieb went on to say, “. . .prescribers can have confidence in the drug’s uniform strength and consistent delivery that support appropriate dosing needed for treating patients with these complex and serious epilepsy syndromes. We’ll continue to support rigorous scientific research on the potential medical uses of marijuana-derived products. . . But, at the same time, we are prepared to take action when we see the illegal marketing of CBD-containing products with serious, unproven medical claims (that) can keep patients from accessing appropriate, recognized therapies to treat serious and even fatal diseases.”

“Today’s approval of Epidiolex is a historic milestone, offering patients and their families the first and only FDA-approved CBD medicine to treat 2 severe, childhood-onset epilepsies,” said Justin Gover, GW’s Chief Executive Officer. “This approval is the culmination of GW’s many years of partnership with patients, their families, and physicians in the epilepsy community to develop a much needed, novel medicine. These patients deserve and will soon have access to a cannabinoid medicine that has been thoroughly studied in clinical trials, manufactured to assure quality and consistency, and available by prescription under a physician’s care.”